Research for Rare - Research for rare diseases

GAIN - German genetic multi-organ Auto-Immunity Network

What are multi-organ autoimmune diseases?

Multi-organ autoimmune diseases belong to the “ultra-rare” disorders. In this case, the body’s own immune system mistakenly attacks its own organs. Affected individuals then develop inflammation of several organs, for example the bone marrow, intestines, lungs, kidneys, skin and nervous system. Due to the rarity and complexity of the disease patterns, it often takes a long time before the correct diagnosis is made.
Single genes and monogenetic mutations have already been discovered as the cause of this group of diseases. These and other findings also help to improve the understanding and treatment for more common polygenic autoimmune diseases.

Joint Research in the GAIN network

In the GAIN research network, experts at various German locations are working together to research the causes and therapies of genetic multi-organ autoimmune diseases. The Federal Ministry of Education and Research (BMBF) has been funding the network since 2019.

In order to facilitate diagnosis and counseling of affected individuals and their families and to improve treatment, the individual research teams are looking into the underlying molecular and cellular pathomechanisms of individual diseases as well as possible molecular interventions as a therapeutic option. In this context, already known but also new genetic causes of disease are analyzed comprehensively. Samples from a biomaterial bank are already available to the researchers for their work. Another focus is on the development of a uniform approach to the identification, diagnosis and treatment of multi-organ autoimmune diseases.

Available data are systematically collected in a patient registry. Through this registry, patients could be recruited for a companion clinical trial investigating the safety and efficacy of the immune-modulatory drug Abatacept in a first funding period (2019-2022). In the second funding period (2023-2025), the quality of life of patients will now be investigated with questionnaires and patient involvement in order to better address their everyday problems.

Experts of GAIN are actively involved in the European Reference Network on Rare Primary Immunodeficiency, AuToinflammatory and Autoimmune diseases (ERN-RITA).

Projects
Contact

Prof. Dr. med Bodo Grimbacher
Medical Center – University of Freiburg
Institute for Immunodeficiency,
Breisacher Str. 115
79106 Freiburg i. Br.
Phone +49 761 27077-732
Email: bodo.grimbacher@uniklinik.freiburg.de
Website: www.g-a-i-n.de

Publications

Research on Rare Diseases in Germany – The GAIN Registry: a registry for individuals with congenital multi-organ autoimmune diseases – Journal of Health Monitoring 4/2023

More information

CLINICAL TRIALS GAIN

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